We were made to give people affected by a rare genetic condition an answer – treatment for their condition. Rare genetic conditions are not statistically relevant when considered individually, but the same can’t be said about a person’s life.

We support the best projects chosen using a rigorous and transparent selection process, following consultation with an internationally-renowned committee of qualified scientists.

We fund research to develop effective therapies that can radically improve patients’ quality of life, to identify diseases that are as rare as they are unknown, and bring us closer every day to a cure.